Question

What is the permanent cure of adenosine deaminase (ADA) deficiency in children?

• A. Bone marrow transplantation
• B. Enzyme replacement therapy in which functional ADA is given to patient by injection
• C. Infusion of genetically engineered lymphocytes (in which functional ADA-cDNA is introduced) into the patient's blood
• D. Introduction of gene isolated from the bone marrow cells which produce ADA, into the cells of the patient at early embryonic stages

Answer 1

The correct option is D Introduction of gene isolated from the bone marrow cells which produce ADA, into the cells of the patient at early embryonic stages

Adenosine deaminase (ADA) deficiency can be treated by bone marrow transplantation, or by enzyme replacement therapy, in which functional ADA is given to the patient by injection. But permanent treatment can be done by introduction of gene isolated from bone marrow cells, which produces ADA, into the cells of the patient at early embryonic stage.